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Latest Inborn errors of metabolism Stories

2011-06-01 07:47:09

(Ivanhoe Newswire) -- Cells, like ordinary households, produce "garbage" "“ debris and dysfunctional elements "“ that need disposal. When the mechanism for taking out this garbage fails, rare genetic diseases called lysosomal storage disorders (including Tay-Sachs, Batten and Fabry disease) can disable and even kill the children they affect. In adults, such failure leads to neurodegenerative diseases that occur later in life, such as Alzheimer's and Parkinson's diseases. An...

2011-05-27 13:31:15

Cells, like ordinary households, produce "garbage" "“ debris and dysfunctional elements - that need disposal. When the mechanism for taking out this garbage fails, rare genetic diseases called lysosomal storage disorders (including Tay-Sachs, Batten and Fabry disease) can disable and even kill the children they affect. In adults, such failure leads to neurodegenerative diseases that occur later in life, such as Alzheimer's and Parkinson's diseases. An international partnership between...

2011-05-17 13:32:00

WEINHEIM, Germany, May 17, 2011 /PRNewswire/ -- International biotechnology firm Cytonet, which is investigating a liver cell infusion of donated human liver cells for the treatment of urea cycle disorders (UCD) in young children, announced it has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). "This designation recognizes our work to advance the landscape of UCD treatment for young children and offer a much-needed new option for a very serious...

2011-05-04 15:01:00

CRANBURY, N.J., May 4, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD), a biopharmaceutical company at the forefront of developing therapies for rare diseases, today announced financial results for the first quarter ended March 31, 2011. The Company also highlighted recent and upcoming milestones, including an update on patient enrollment in the ongoing Phase 3 study of its lead program Amigal(TM) (migalastat HCl) for Fabry disease. Development Pipeline Highlights...

2011-04-26 00:00:27

Children's Memorial Hospital in Chicago is one of only 12 sites across the United States enrolling patients in a national clinical research trial which, for the first time is the United States, is using an investigational liver cell infusion to treat urea cycle disorders (UCDs) in children. UCDs are congenital and often life-threatening disorders of ammonia metabolism in the liver. Neurotoxic ammonia accumulates in the body, which may lead "“ depending on the severity of the disease...

2011-04-14 14:48:00

WEINHEIM, Germany, April 14, 2011 /PRNewswire/ -- International biotechnology firm Cytonet recently launched the first ever clinical trial in the United States using an investigational liver cell infusion to treat urea cycle disorders (UCD) in children. The first patient in the Phase II trial, which will seek to enroll 20 patients in 12 medical centers, is a child with citrullinemia, which is a hereditary UCD. He received six liver cell infusions on six consecutive days at Yale School of...

2011-04-11 15:50:47

Study focused on ER visits or hospitalizations in children with inborn errors of metabolism Children with inborn errors of metabolism received vaccines on the same immunization schedule as did healthy infants, according to Kaiser Permanente Vaccine Study Center scientists who examined the Kaiser Permanente Northern California population. In addition, immunization was not associated with significant increases in emergency room visits or hospitalizations during the month following vaccination,...

2011-04-07 07:00:00

SAN DIEGO, April 7, 2011 /PRNewswire/ -- Zacharon Pharmaceuticals, Inc. today announced that the company has entered into a strategic research collaboration with Pfizer Inc. to develop drugs for orphan diseases, including lysosomal storage disorders. The potential value of the collaboration to Zacharon is approximately USD $210 million. The collaboration includes the potential development of compounds that may be discovered using Zacharon's innovative platform for developing small molecule...

2011-03-08 06:00:00

CRANBURY, N.J., March 8, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND). AT2220 is a pharmacological chaperone in development as a treatment for Pompe disease. Based on data provided by Amicus, the FDA agreed with the Company's proposal to resume clinical development of AT2220, starting with a new Phase...

2011-02-16 07:30:00

CRANBURY, N.J., Feb. 16, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced that additional positive data from the ongoing Phase 2 extension study of its investigational drug Amigal(TM) (migalastat HCl) for Fabry disease will be presented at the Lysosomal Disease Network WORLD Symposium in Las Vegas, Nevada, February 16-18th, 2011. In addition, the Company announced that it will present encouraging data from its preclinical studies evaluating the co-administration of...


Word of the Day
conjunto
  • A style of popular dance music originating along the border between Texas and Mexico, characterized by the use of accordion, drums, and 12-string bass guitar and traditionally based on polka, waltz, and bolero rhythms.
The word 'conjunto' comes through Spanish, from Latin coniūnctus, past participle of coniungere, to join together; see conjoin